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A study published in the journal Neurology ("Hematopoietic stem cell transplantation in patients with active secondary progressive multiple sclerosis") reported that transplanted patients' own hematopoietic stem cells may delay disability progression
in patients with secondary progressive multiple sclerosis than other anti-inflammatory disease modification therapies (DMTs).
Matilde Inglese, MD, professor of neurology at the University of Genoa in Italy and senior author of the study, said: "Hematopoietic stem cell transplantation has previously been found to delay disability in patients with relapsing-remitting MS, but it is unclear whether this transplantation helps delay disability
in advanced disease.
" "Our results are encouraging because, while there is currently a modest or small benefit in the treatment of SPMS, our study found that stem cell transplantation may not only delay disability over many other MS medications, but may also have a slight improvement
in symptoms.
"
Patients are initially diagnosed with relapsing-remitting multiple sclerosis, alternating periods of active episodes of symptoms, eventually progressing to SPMS, with gradual but steady progression
of the disease.
The exact mechanism leading to increased neurodegeneration in SPMS is unknown, but there is evidence that the primary role of innate and adaptive immune mechanisms is to drive inflammation
in the brain parenchyma, light meninges, and cerebrospinal fluid.
Early research suggests that the use of anti-inflammatory drugs such as siponimod may be beneficial
for SPMS treatment.
However, DMT only modestly reduces the overall risk of disability, and the duration of its effects is unclear
.
AHSCT, on the other hand, resets immune dysfunction
by clearing abnormal immune cells that fight the body's own cells.
Early studies have shown that AHSCT can slow neurodegeneration in SPMS, but controversy remains
.
"In this study, we wanted to assess whether autologous hematopoietic stem cell transplantation prolongs the time
to confirm disability progression compared to other disease-modifying therapies in SPMS," the authors note.
The current retrospective study conducted a 10-year retrospective study of clinical data from 79 SPMS patients who underwent autologous hematopoietic stem cell transplantation (AHSCT) and 1975 SPMS patients who received β-interferon, azathioprine, methotrexate, cyclophosphamide, or other DMTs
.
The ages, sexes, and degrees of disability matched
between the two groups.
Data were collected from the Italian Study Group of Bone Marrow Transplantation and the Italian Multiple Sclerosis Registry
.
The specific objective of the study was to compare the proportion
of SPMS patients treated with AHSCT at 6 months with other DMT-treated patients with disability progression (CDP).
The degree of disability of patients is measured by the Extended Disability Status Scale (EDSS), an established method for quantifying disability, with scores ranging from 0 (asymptomatic) to 10 (death due to MS).
Initially, participants in both groups scored a median score of 6.
5
.
Afterward, when participants occasionally needed to walk about 100 meters using crutches or support, they were given a 6.
0 point with or without rest
.
When patients needed to walk non-stop on both sides for about 20 meters with crutches or braces without rest, they scored 6.
5
.
The researchers found that after 5 years of treatment, 62 percent of SPMS patients with AHSCT did not have a worsening disability, compared with 46 percent of patients taking other DMTs
.
After 5 years of treatment, patients treated with AHSCT were also more likely to see sustained improvement over time, with 19 percent having fewer disabilities than at the start of the study, compared with only 4 percent
of those receiving medication.
Over a decade, disability scores for AHSCT patients decreased by an average of 0.
01 points per year, while the average scores of other DMT patients increased by 0.
16 points
per year.
"Our study shows that hematopoietic stem cell transplantation is associated with
a higher likelihood of slowing disability progression and improving disability compared to other therapies," Inglese said.
"While these results are encouraging, they do not apply to patients
with secondary progressive MS who do not have signs of inflammatory activity.
" We need more studies in larger populations to confirm our findings
.
”
The results of this retrospective and observational study suggest correlation, but do not establish a causal relationship
.
In addition, the study did not include all anti-inflammatory drugs
prescribed by SPMS patients in the DMT group.